AIDS cure finally found?
Wender said that although initial tests are being done on animals, blood from AIDS patients who have been on immunosuppressive therapy is also being used.
“This is hugely exciting. We’re dealing with real cells from real people who have a real problem,” he said.
The National Institutes of Health is funding the research, under which Wender took prostratin from the bark of the mamala tree and bryostatin and has reproduced them in his laboratory for targeted medical uses.
Prostratin is said to flush the HIV virus out of cells where it hides. Previous drugs for HIV and AIDS have killed the virus when it is out in the open, but not when it remains hidden in cell reservoirs. If patients stop taking their medications, the virus quickly comes out of hiding and begins to reproduce.
Wender recreated prostratin and designed new analogs of the compound. Now, prostratin is 100 times more powerful than when it was naturally contained in the mamala tree.
Prostratin is being developed by the AIDS Research Alliance (ARA), a non-profit organization in West Hollywood, Calif. searching for a cure for AIDS. The ARA has pledged to ensure access to the drug for people with AIDS in developing countries after it obtains approval from the U.S. Food and Drug Administration (FDA) for human use.
Brown, the medical director of the ARA, told Healthline the alliance is about two-thirds of the way through experiments required to bring prostratin to market.
“It’s a slow slog because we’re a non-profit,” Brown said. “I think there's more interest on the part of pharmaceutical companies. When we first started, everyone thought we were crazy.”
Wender’s work goes beyond a potential cure for AIDS and can be potentially used in therapies for cancer and Alzheimer’s disease as well.
Some AIDS-cure hopes rest on the so-called DNA scissors.
Scientists claim that some people are highly resistant or even immune to HIV, the virus that causes AIDS, and when they looked at these people’s DNA, they found that many of them shared a specific genetic mutation called CCR5 delta32. This mutation inactivates the CCR5 gene, i.e. these people make no CCR5 protein, which means they are highly resistant to getting AIDS because the most virulent form of the virus, HIV-1, needs it to get into blood cells.
Learning about this mutation, scientists worked on a new class of drugs, CCR5 receptor antagonists. These drugs block the action of the CCR5 protein making it very difficult for HIV-1 to enter the patient’s blood cells. At least one of these, Selzentry, is currently being used to treat some cases of HIV.
Unfortunately, scientists can’t do this for everyone with AIDS, so
one way would be to engineer the mutation into a patient’s blood cells. Researchers from Sangamo Biosciences funded by California Stem Cell Bond have come up with a product called SB-728 that can specifically inactivate the CCR5 gene in human cells. This treatment has even made it into clinical trials where so far the results appear to be encouraging.
However, this specific treatment could still fail in ongoing clinical trials or even turn out to not be a permanent cure and then the scissors come into play.
The new cure is a pair of molecular scissors that will cut human DNA only at the CCR5 gene. Once cut, cells will wreck the gene in the process of repairing the DNA. A broken CCR5 gene means no CCR5 protein, which means no HIV-1 infection.
The scientists at Sangamo whipped up a protein that could specifically recognize the CCR5 gene and cut the CCR5 gene in cells in the lab and in clinical trials. The new method is undergoing clinical tests.
Meanwhile, Canadian researchers claim a vaccine to prevent HIV has aced clinical testing.
The team from the Schulich School of Medicine & Dentistry at Western University said the first phase of testing not only succeeded, but boosted the production of antibodies in patients it was tested on.
SAV001 HIV vaccine is based on a genetically modified “dead” version of the virus.
“We infect the cells with a genetically modified HIV-1,” team leader Dr. Chil-Yong Kang told Ontario Business Report. “The infected cells produce lots of virus, which we collect, purify and inactivate so that the vaccine won’t cause AIDS in recipients, but will trigger immune responses.”
U.S. clinical testing began in the in March 2012, looking at HIV-infected men and women between the ages of 18 and 50. Half the target group was provided with a placebo, while the other group was given SAV001. The first phase of trials wrapped up last month, with researchers optimistic about the vaccine’s future.
Sumagen, the South Korean biotech firm sponsoring the vaccine, cited manufacturing, as well as USFDA requirements as hurdles in bringing the vaccine to market, but, if all goes well in trials, it could be commercially available in five years.
“We are now prepared to take the next steps towards Phase II and Phase III clinical trials,” CEO Jung-Gee Cho said in a press release.
“We are opening the gate to pharmaceutical companies, government, and charity organization for collaboration to be one step closer to the first commercialized HIV vaccine.”
So, we need to wait to see what happens next. These trials may not only lead to a functional cure for AIDS, but they could also open up a whole new way to treat other diseases.